ENA oligonucleotides as therapeutics
1Core Technology Research Laboratories, Sankyo Co Ltd, 1-2-58 Hiromachi, Shinagawa-ku, Tokyo 140-8710, Japan. koizum@sankyo.co.jp
Abstract:
Oligonucleotides containing 2'-O,4'-C-ethylene-bridged nucleic acid (ENA) residues have two notable properties: (i) considerable affinity to complementary single-stranded RNA and double-stranded DNA; and (ii) dramatically high resistance against nucleases. On the basis of these properties, the in vitro and in vivo applications of ENA for gene silencing as antisense oligonucleotides, triplex-forming oligonucleotides and small interfering RNAs are discussed here. ENA oligonucleotides also have potential as therapeutic agents for Duchenne muscular dystrophy by a mechanism of exon skipping.
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